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論文リスト

doi title author source_title year
261 10.1002/acn3.779 Neurofilament As A Potential Biomarker For Spinal Muscular Atrophy Darras, Basil T.; Crawford, Thomas O.; Finkel, Richard S.; Mercuri, Eugenio; De Vivo, Darryl C.; Oskoui, Maryam; Tizzano, Eduardo F.; Ryan, Monique M.; Muntoni, Francesco; Zhao, Guolin; Staropoli, John; Mccampbell, Alexander; Petrillo, Marco; Stebbins, Christopher; Fradette, Stephanie; Farwell, Wildon; Sumner, Charlotte J. Annals Of Clinical And Translational Neurology 2019
2184 10.1002/ppul.24203 Pediatric Pulmonology 2018
2205 10.1002/prp2.447 Risdiplam Distributes And Increases Smn Protein In Both The Central Nervous System And Peripheral Organs Poirier, Agnes, 0000-0002-7077-009X; Weetall, Marla; Heinig, Katja; Bucheli, Franz; Schoenlein, Kerstin; Alsenz, Jochem; Bassett, Simon; Ullah, Mohammed; Senn, Claudia; Ratni, Hasane; Naryshkin, Nikolai; Paushkin, Sergey; Mueller, Lutz Pharmacology Research & Perspectives 2018
2511 10.1007/s00415-019-09389-8 Nfl Is A Marker Of Treatment Response In Children With Sma Treated With Nusinersen Olsson, Bob; Alberg, Lars; Cullen, Nicholas C.; Michael, Eva; Wahlgren, Lisa; Kroksmark, Anna-Karin; Rostasy, Kevin; Blennow, Kaj; Zetterberg, Henrik; Tulinius, Mar Journal Of Neurology 2019
8686 10.1016/j.nmd.2019.09.007 Nusinersen Initiated In Infants During The Presymptomatic Stage Of Spinal Muscular Atrophy: Interim Efficacy And Safety Results From The Phase 2 Nurture Study De Vivo, Darryl C.; Bertini, Enrico; Swoboda, Kathryn J.; Hwu, Wuh-Liang; Crawford, Thomas O.; Finkel, Richard S.; Kirschner, Janbernd; Kuntz, Nancy L.; Parsons, Julie A.; Ryan, Monique M.; Butterfield, Russell J.; Topaloglu, Haluk; Ben-Omran, Tawfeg; Sansone, Valeria A.; Jong, Yuh-Jyh; Shu, Francy; Staropoli, John F.; Kerr, Douglas; Sandrock, Alfred W.; Stebbins, Christopher; Petrillo, Marco; Braley, Gabriel; Johnson, Kristina; Foster, Richard; Gheuens, Sarah; Bhan, Ishir; Reyna, Sandra P.; Fradette, Stephanie; Farwell, Wildon Neuromuscular Disorders 2019
8787 10.1016/j.pediatrneurol.2019.05.005 Impact Of Age And Motor Function In A Phase 1/2A Study Of Infants With Sma Type 1 Receiving Single-Dose Gene Replacement Therapy Lowes, Linda P.; Alfano, Lindsay N.; Arnold, W. David; Shell, Richard; Prior, Thomas W.; Mccolly, Markus; Lehman, Kelly J.; Church, Kathleen; Sproule, Douglas M.; Nagendran, Sukumar; Menier, Melissa; Feltner, Douglas E.; Wells, Courtney; Kissel, John T.; Al-Zaidy, Samiah; Mendell, Jerry Pediatric Neurology 2019
10397 10.1016/s1474-4422(20)30037-5 Nusinersen In Adults With 5Q Spinal Muscular Atrophy: A Non-Interventional, Multicentre, Observational Cohort Study Hagenacker, Tim; Wurster, Claudia D; Gunther, Rene; Schreiber-Katz, Olivia; Osmanovic, Alma; Petri, Susanne; Weiler, Markus; Ziegler, Andreas; Kuttler, Josua; Koch, Jan C; Schneider, Ilka; Wunderlich, Gilbert; Schloss, Natalie; Lehmann, Helmar C; Cordts, Isabell; Deschauer, Marcus; Lingor, Paul; Kamm, Christoph; Stolte, Benjamin; Pietruck, Lena; Totzeck, Andreas; Kizina, Kathrin; Monninghoff, Christoph; Von Velsen, Otgonzul; Ose, Claudia; Reichmann, Heinz; Forsting, Michael; Pechmann, Astrid; Kirschner, Janbernd; Ludolph, Albert C; Hermann, Andreas; Kleinschnitz, Christoph The Lancet Neurology 2020
11058 10.1021/acs.jmedchem.8b00741 Discovery Of Risdiplam, A Selective Survival Of Motor Neuron-2 (Smn2) Gene Splicing Modifier For The Treatment Of Spinal Muscular Atrophy (Sma) Ratni, Hasane, 0000-0002-8151-8295; Ebeling, Martin; Baird, John; Bendels, Stefanie; Bylund, Johan; Chen, Karen S.; Denk, Nora; Feng, Zhihua; Green, Luke; Guerard, Melanie; Jablonski, Philippe; Jacobsen, Bjoern; Khwaja, Omar; Kletzl, Heidemarie; Ko, Chien-Ping; Kustermann, Stefan; Marquet, Anne; Metzger, Friedrich; Mueller, Barbara; Naryshkin, Nikolai A.; Paushkin, Sergey V.; Pinard, Emmanuel; Poirier, Agnes; Reutlinger, Michael; Weetall, Marla; Zeller, Andreas; Zhao, Xin; Mueller, Lutz Journal Of Medicinal Chemistry 2018
11060 10.1021/acs.jmedchem.8b01291 Discovery Of Small Molecule Splicing Modulators Of Survival Motor Neuron-2 (Smn2) For The Treatment Of Spinal Muscular Atrophy (Sma) Journal Of Medicinal Chemistry 2018
19750 10.1172/jci124120 Age-Dependent Smn Expression In Disease-Relevant Tissue And Implications For Sma Treatment Journal Of Clinical Investigation 2019
20384 10.1212/wnl.0000000000007527 Nusinersen In Later-Onset Spinal Muscular Atrophy Darras, Basil T.; Chiriboga, Claudia A.; Iannaccone, Susan T.; Swoboda, Kathryn J.; Montes, Jacqueline; Mignon, Laurence; Xia, Shuting; Bennett, C. Frank; Bishop, Kathie M.; Shefner, Jeremy M.; Green, Allison M.; Sun, Peng; Bhan, Ishir; Gheuens, Sarah; Schneider, Eugene; Farwell, Wildon; De Vivo, Darryl C. Neurology 2019
20933 10.3233/jnd-190403 Avxs-101 (Onasemnogene Abeparvovec) For Sma1: Comparative Study With A Prospective Natural History Cohort Journal Of Neuromuscular Diseases 2019
20934 10.3233/jnd-190416 Safety And Treatment Effects Of Nusinersen In Longstanding Adult 5Q-Sma Type 3 <U+2013> A Prospective Observational Study Walter, Maggie C.; Wenninger, Stephan; Thiele, Simone; Stauber, Julia; Hiebeler, Miriam; Greckl, Eva; Stahl, Kristina; Pechmann, Astrid; Lochmuller, Hanns; Kirschner, Janbernd; Schoser, Benedikt Journal Of Neuromuscular Diseases 2019
20935 10.3233/jnd-190428 One Year Of Newborn Screening For Sma <U+2013> Results Of A German Pilot Project Vill, Katharina; Kolbel, Heike; Schwartz, Oliver; Blaschek, Astrid; Olgemoller, Bernhard; Harms, Erik; Burggraf, Siegfried; Roschinger, Wulf; Durner, Jurgen; Glaser, Dieter; Nennstiel, Uta; Wirth, Brunhilde; Schara, Ulrike; Jensen, Beate; Becker, Marc; Hohenfellner, Katharina; Muller-Felber, Wolfgang Journal Of Neuromuscular Diseases 2019